NCT05035862

Mechanisms of Interferon Gamma-primed Mesenchymal Stromal Cells (MSCs) for Moderate-to-severe Persistent Asthma

A Phase 1 Study to Evaluate Safety, Toxicity, and Potential Mechanisms of Interferon Gamma-primed Mesenchymal Stromal Cells (MSCs) for Moderate-to-severe Persistent Asthma

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Emory University · Academic / Other
• Sex: All
• Age: 18 Years – 30 Years
• Healthy volunteers: Not accepted

Summary

This study is a two strata, dose escalation Phase I clinical trial designed to assess the safety and determine the maximal tolerated dose (MTD) of allogenic cord tissue derived MSCs (cMSCs, stratum 1) and allogeneic, interferon-γ primed bone marrow MSCs (γMSCs, stratum 2). Each stratum is designed to independently accrue 3 children at a dose level 1 of 2x106 cells/kg and 6 children at dose level 2 of 10x106 cells/kg, resulting in 9 children in each stratum. The primary objectives are to determine the safety and toxicity of allogeneic cord tissue derived MSCs and allogeneic interferon-γ primed bone marrow derived MSCs.

Detailed description

Asthma affects one out of every 10 patients in the United States. Many of these patients have poor asthma symptom control. For example, patients with moderate-to-severe persistent asthma have ongoing symptoms and airway inflammation despite aggressive treatment with asthma medications. These patients are at increased risk for medication-related side effects and potentially life-threatening exacerbations. Novel therapies are critically needed for this population. Mesenchymal stem cells (MSCs) are cells that reside in the bone marrow. MSCs are anti-inflammatory and also promote body tissue repair. This study will determine whether one form of MSCs called "interferon gamma-primed MSCs or γMSCs" are safe for patients with moderate-to-severe asthma. Patients will receive a single intravenous infusion of γMSCs at either 2x10\^6 cells/kg or 5x10\^6 cells/kg. Up to 12 young adults will be enrolled. The total sample size will not exceed 24 participants. The study will take place at Children's Healthcare of Atlanta (for patient activities) and at Emory University (for laboratory research activities). Participants will be identified from the asthma clinics at Children's Healthcare of Atlanta. Participants will complete up to 12 visits over 1 year and will be compensated for their time and travel. At the completion of the study, any samples remaining after experimentation will be de-identified and made available for future research. While some study participants may receive no direct benefit from participating in this study, others may benefit from the close monitoring of their respiratory health, specialized asthma education, and general evaluation of their condition, including lung function tests. Some participants also achieve psychological benefit from participating in an important research study and from interaction with the study staff. It is also possible that the knowledge obtained from this study, such as identification of biomarkers, may assist in the creation of novel asthma therapies in the future.

Conditions

• Asthma

Interventions

Type	Name	Description
DRUG	Albuterol Sulfate	Participants who experience symptoms of cough, dyspnea, chest tightness or wheezing will initiate use of albuterol (2 inhalations, 90 mcg/actuation) by metered dose inhaler (MDI) every 20 minutes for up to 1 hour and then every 4 hours if necessary.
DRUG	Interferon gamma-primed mesenchymal stromal cells (MSCs)	IFNγ-primed bone marrow MSCs at a dose level of 2x106 cells/kg and a dose level of 5x106 cells/kg
DRUG	Prednisone	Prednisone is recommended if the participant uses more than 12 inhalations of albuterol in 24 hours (excluding preventive use before exercise), or if the patient has ongoing symptoms for 48 hours or longer. The recommended prednisone dose for acute exacerbations is 2 mg/kg/day (maximum 60 mg) as a single dose for two days followed by 1 mg/kg/day (maximum 30 mg) as a single dose for two days. All administered doses will be rounded down to the nearest 10 mg.

Timeline

• Start date: 2022-03-16
• Primary completion: 2022-12-16
• Completion: 2022-12-16
• First posted: 2021-09-05
• Last updated: 2025-06-03

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study